| abstract |
A composition for use in the treatment of Pompe disease, wherein the composition comprises a rAAV2 / 8 vector, wherein the rAAV2 / 8 vector comprises a heterologous nucleic acid molecule encoding acid α-glucosidase, wherein the heterologous nucleic acid is operatively linked to a desmin promoter, a myocyte 2-specific enhancer factor enhancer element, and a MyoD enhancer element, wherein the composition is administered to a subject in need, by intramuscular, intrathoracic injection , intraspinal, intrathecal, intracisternal or intravenous. |